EUCTR2015-001985-25-ES进行中（未招募）1 期
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 250 in Patients with Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 33
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •Part 1: Dose Escalation Period:
- •Has deficient NAGLU enzyme activity at screening.
- •Is between 1 and 10 years of age.
- •Has presented with signs/symptoms consistent with MPS IIIB; for individuals who have not presented with signs/symptoms of the disease (e.g., siblings of known patients), the determination of eligibility will be at the discretion of the BioMarin medical monitor in conjunction with the site investigator.
- •Written informed consent from parent or legal guardian and assent from subject, if required.
- •Has the ability to comply with protocol requirements, in the opinion of the investigator.
- •Part 2: Stable Dose Period:
- •Participated in and met protocol requirements for transitioning from Study 250-901 or participated in Part 1 of Study 250-201
- •Written informed consent from parent or legal guardian and assent from subject, if required
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 33
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Part 1: Dose Escalation Phase:
- •Has received stem cell, gene therapy, or enzyme replacement therapy for MPS IIIB.
- •Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities).
- •Has contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain).
- •Has a history of poorly controlled seizure disorder.
- •Is prone to complications from intraventricular drug administration, including patients with hydrocephalus or ventricular shunts.
- •Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study.
- •Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with protocol requirements, the subject's well-being or safety, or the interpretability of the subject's clinical data.
- •Is pregnant at any time during the study
- •Part 2: Stable Dose Period:
- •Has received stem cell, gene therapy or ERT for MPS IIIB
- •Has contraindications for neurosurgery (e.g., congenital heart disease, severe respiratory impairment, or clotting abnormalities)
- •Has contraindications for MRI scans (e.g., cardiac pacemaker, metal fragment or chip in the eye, or aneurysm clip in the brain)
- •Is prone to complications from intraventricular drug administration, including patients with hydrocephalus or ventricular shunts
- •Has received any investigational medication within 30 days prior to the Baseline visit or is scheduled to receive any investigational drug during the course of the study
- •Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject´s ability to comply with protocol requirements, the subject´s well-being or safety, or the interpretability of the subject´s clinical data.
- •Is pregnant at any time during the study
研究者
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